Supporting Innovation in Friedrich Ataxia Clinical Research

Doherty Clinical Trials are excited to share the recent announcement in positive biomarker and clinical data results as part of the ongoing Phase 1/2 RESTORE-FA trial, in collaboration with Design Therapeutics.

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The trial is evaluating DT-216P2, an investigational therapy for patients with Friedreich ataxia, a rare and progressive neurodegenerative disease with significant unmet medical need.

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Friedreich ataxia causes progressive damage to the nervous system resulting in symptoms which include weakness, loss of balance and coordination (ataxia), speech problems and heart disease.

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The DT-261P2 therapy being investigated is a GeneTAC ® small-molecule therapeutic candidate designed to increase frataxin (FXN) expression by targeting the genetic root cause of Friedreich ataxia.

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Design Therapeutics recently announced positive data from the 4-week IV cohorts in the trial, reporting dose-dependent improvements in multiple clinical outcome measures alongside increases in endogenous frataxin mRNA and protein levels.

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Dr Tom Frisby, Lead Sub-Investigator at DCT, said the emerging data highlighted the importance of continued investment in early-phase clinical research for rare diseases. “Friedreich ataxia patients and families urgently need new treatment options. It is encouraging to see early clinical data demonstrating both biological activity and potential clinical benefit with this novel drug candidate. We are proud to contribute to research that may help advance future therapies for patients living with this devastating condition.”

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Chris Storgard, Chief Medical Officer at Design Therapeutics said: “We are encouraged by these findings and grateful for [Doherty Clinical Trials] dedication and continued commitment to this program.”

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Doherty Clinical Trials would like to thank the study participants and families, Design Therapeutics, our investigator and clinical teams, and the broader clinical research team for their ongoing commitment to advancing innovative therapies in rare disease research.

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To learn more about the results, visit the Design Therapeutics page here.

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